Allogeneic Stem Cell Transplant With Alpha/Beta T AND B Cell Depletion for Hematologic Malignancies

Phase 2

Active, not recruiting

• Conditions: Myelodysplasia; Leukemia; Lymphoma
• Interventions: Device: Alpha Beta T cell depletion

• Registration Number: NCT02323867
• Lead Sponsor: Children's Hospital of Philadelphia

Brief Summary

This is a single arm pilot study for patients using α/β T cell-depleted peripheral Stem Cell Transplantation (PSCT) in with alternative donor sources with hematologic malignancies receiving alternative donor (unrelated or partially matched related) mobilized peripheral stem cells (PSCs) using the CliniMACS system for T cell depletion plus CD19+ B cell depletion to determine efficacy as determined by engraftment and graft versus host disease (GVHD), and one year leukemia free survival.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-10
• Study First Submitted: 2014-10-22
• Study First Submitted QC: 2014-12-23
• Study First Posted: 2014-12-24
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-31
• Last Update Posted: 2025-01-01
• Primary Completion: 2025-10
• Study Completion: 2026-10

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 140

Inclusion Criteria

1. Leukemias/lymphomas

   • Acute myeloid leukemia, primary or secondary Disease status: remission or <10% bone marrow blasts
   • Myelodysplasia
   • Acute lymphoblastic leukemia Disease status: in hematologic remission
   • Chronic myelogenous leukemia Disease status: chronic phase, accelerated phase or blast crisis now in second chronic phase.
   • Mixed lineage or biphenotypic acute leukemia
   • Lymphoblastic lymphoma Disease status: remission
   • Burkitt's lymphoma/leukemia: Disease status- in remission

2. Acceptable organ status clearance

3. No active untreated infection. Patients with possible fungal infections must have had at least 2 weeks of appropriate anti-fungal antibiotics and be asymptomatic

4. Signed informed consent.

5. Negative pregnancy test for females of childbearing potential.

Exclusion Criteria

1. Patients who do not meet disease, organ or infectious criteria.
2. No suitable donor
3. Received previous allogeneic transplant

Donor must be either unrelated, mismatched up to one antigen/one antigen and one allele or a related, HLA match other than a sibling.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Alpha Beta Non-irradiation regimen	Alpha Beta T cell depletion	Alpha Beta Non-irradiation regimen Day Treatment * 9 Busulfan + ATG * 8 Busulfan + ATG * 7 Busulfan +ATG * 6 Busulfan * 5 Thiotepa * 4 Thiotepa * 3 Cyclophosphamide * 2 Cyclophosphamide * 1 0 Transplant with alpha beta T cell depleted stem cells
Alpha Beta Total Body Irradiation - total body irradiation (TBI) first	Alpha Beta T cell depletion	Alpha Beta Total Body Irradiation - TBI first Day Treatment * 11 Anti-thymocyte globulin (ATG) * 10 ATG * 9 ATG * 8 TBI * 7 TBI * 6 TBI * 5 Thiotepa * 4 Thiotepa * 3 Cyclophosphamide * 2 Cyclophosphamide * 1 Rest 0 Transplant with alpha beta T cell depleted stem cells
Alpha Beta Total Body Irradiation - TBI last	Alpha Beta T cell depletion	Alpha Beta Total Body Irradiation - TBI last Day Treatment * 9 ATG * 8 ATG * 7 Thiotepa + ATG * 6 Thiotepa * 5 Cyclophosphamide * 4 Cyclophosphamide * 3 TBI * 2 TBI * 1 TBI 0 Transplant with alpha beta T cell depleted stem cells

Primary Outcome Measures

Name	Time	Method
Engraftment: rates of engraftment, defined as the first day of Absolute Neutrophil Count (ANC) greater than 500 for the first of three consecutive days	1 year

Secondary Outcome Measures

Name	Time	Method
Severe acute and chronic graft versus host disease (GVHD)	1 year	Estimation of the incidence and extent of acute and chronic GVHD. The overall incidence of acute GVHD, the incidence of Grade 4 acute GVHD and the incidence of chronic GVHD.

Trial Locations

Locations (1)

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States